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ClinicalTrials.gov public records Last synced May 21, 2026, 8:08 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Gene Transfer Clear all
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Methylmalonic Acidemia
Interventions
hLB-001
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2037
U.S. locations
4
States / cities
Atlanta, Georgia • Pittsburgh, Pennsylvania • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed Early Phase 1 Interventional
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
rAAV2-CB-hAAT Gene Vector
Genetic
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2020
U.S. locations
2
States / cities
Gainesville, Florida • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 9, 2020 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Hemophilia A
Interventions
AAV2/8-HLP-FVIII-V3
Biological
Lead sponsor
University College, London
Other
Eligibility
18 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
3
States / cities
Boise, Idaho • Lexington, Kentucky • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 4, 2025 · Synced May 21, 2026, 8:08 PM EDT
Withdrawn Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Methylmalonic Acidemia (MMA)
Interventions
SEL-302
Drug
Lead sponsor
Selecta Biosciences, Inc.
Industry
Eligibility
3 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced May 21, 2026, 8:08 PM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
HIV
Interventions
Antiretroviral Therapy Interruption(ATI)
Other
Lead sponsor
American Gene Technologies International Inc.
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 1, 2022 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Breast Neoplasms, Neoplasm Metastasis
Interventions
peripheral blood progenitor cells carrying MDR1
Genetic
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Alzheimer Disease
Interventions
LX1001
Biological
Lead sponsor
Lexeo Therapeutics
Industry
Eligibility
50 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
2
States / cities
Orlando, Florida • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 30, 2025 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Gyrate Atrophy
Interventions
Gene therapy
Procedure
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Melanoma
Interventions
Talimogene Laherparepvec
Drug
Lead sponsor
BioVex Limited
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
7
States / cities
La Jolla, California • Los Angeles, California • Aurora, Colorado + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2015 · Synced May 21, 2026, 8:08 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011QQ
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Orange, California • Vallejo, California
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Hemophilia A
Interventions
SB-525 (PF-07055480)
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
22
States / cities
Little Rock, Arkansas • Duarte, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Leukemia, B-cell, Lymphoma, Hodgkins, Lymphoma, Non-hodgkins, Lymphoma, B-Cell
Interventions
Allogeneic stem cell transplant, Anti-CD19-chimeric-antigen-receptor-transduced T cells, Leukapheresis
Procedure · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 75 Years
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2024
U.S. locations
2
States / cities
Bethesda, Maryland • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Neoplasms
Interventions
Telephonic interview
Other
Lead sponsor
GlaxoSmithKline
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Collegeville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 4, 2019 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Merkel Cell Carcinoma, Cutaneous Squamous Cell Carcinoma, Non-Melanoma Skin Cancers
Interventions
IFx-Hu2.0
Biological
Lead sponsor
TuHURA Biosciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
4
States / cities
Los Angeles, California • Tampa, Florida • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2024 · Synced May 21, 2026, 8:08 PM EDT
Recruiting Early Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Healthy Adults
Interventions
Follistatin and klotho gene therapy
Genetic
Lead sponsor
Minicircle
Industry
Eligibility
50 Years to 80 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
Conditions
Breast Cancer
Interventions
Autologous, Lethally Irradiated Breast Cancer Cells
Biological
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2021
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 31, 2022 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Krabbe Disease
Interventions
FBX-101
Biological
Lead sponsor
Forge Biologics, Inc
Industry
Eligibility
1 Day to 12 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jan 29, 2025 · Synced May 21, 2026, 8:08 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Metastatic Cancer
Interventions
in vitro tumor reactive, chimeric T cell receptor (CAR ) gene-transduced PBL plus IV aldesleukin, Cyclophosphamide, Fludarabine, Mesna
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2015 · Synced May 21, 2026, 8:08 PM EDT
Recruiting Phase 2 Interventional
View directory record Official record
Conditions
Grade 2 and 3 Late Xerostomia Caused by Radiotherapy for Cancers of the Upper Aerodigestive Tract, Excluding the Parotid Glands
Interventions
AAV2-hAQP1 Concentration 1, AAV2-hAQP1 Concentration 2, Placebo, AAV2-hAQP1 Concentration 3, AAV2-hAQP1 Concentration 4
Genetic · Other
Lead sponsor
MeiraGTx, LLC
Industry
Eligibility
18 Years and older
Enrollment
276 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
18
States / cities
Gilbert, Arizona • Duarte, California • Miami, Florida + 14 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2026 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Sarcoma
Interventions
Autologous HER2-specific T cells, Fludarabine, Cyclophosphamide, Autologous CAR Positive T cells
Genetic · Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2032
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
HIV
Interventions
SB-728mR-HSPC Infusion 3 days following busulfan conditioning
Genetic
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
5
States / cities
Duarte, California • Los Angeles, California • San Francisco, California + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced May 21, 2026, 8:08 PM EDT
Completed Not applicable Interventional
View directory record Official record
Conditions
Malignant Melanoma
Interventions
IL-12p DNA, Intratumoral Electroporation
Biological · Procedure
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Feb 22, 2017 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Fanconi's Anemia, Pancytopenia
Interventions
Transduced CD34+ Cells
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
5 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced May 21, 2026, 8:08 PM EDT